The Cambridge, MA-based biotech company (NASDAQ: VRTX), which has significant operations in San Diego, is announcing today that a combination of standard treatment and its novel drug for hepatitis C was able to cure two-thirds of patients who had failed to respond to a prior round of the standard drugs alone. That response rate for patients on Vertex’s telaprevir (65 percent) far exceeded that of the comparison group, in which just 17 percent of patients were cured after getting the usual combination of pegylated interferon alpha and ribavirin. Vertex is making the announcement today, based on findings from more than 660 patients who enrolled in a study called “Realize.”

“The Realize data represent a major milestone in the development of new treatments for hepatitis C,” Stefan Zeuzem, a professor of medicine at the JW Goethe University Hospital in Frankfurt, Germany and principal investigator of the trial, said in a Vertex statement. “These results may provide hope to people who have not been cured and who are in need of new treatment options.”

This is the third major batch of study results from Vertex this year, and part of its quest to shake up the standard of care for patients with hepatitis C, a chronic liver disease. The company showed back in May, in a study of more than 1,000 patients, that about three-fourths of people getting their first round of treatment were considered cured after getting telaprevir in combination with the standard meds. A second study, released last month, showed that the drug could cut the treatment time in half, which is important because it means patients don’t have to endure the flu-like symptoms caused by the other drugs in the regimen for nearly as long. And today’s announcement reinforces findings from small trials that says the telaprevir-based regimen has far greater ability to kill the virus in the toughest patients to treat.

The business opportunity, which we’ve written about a lot in these pages, is huge. An estimated 6 million people in the U.S. and Europe have chronic hepatitis C infections, and an estimated 650,000 of them have failed a prior round of the standard treatment. If the FDA clears the drug for sale based on the latest clinical trials, telaprevir could generate more than $2.6 billion in U.S. sales by 2013, according to analyst Rachel McMinn of Cowen & Co.

Based on the results of the three pivotal trials, Vertex plans to file an application for FDA approval by the end of this year.

The drug’s side effect profile appeared to be similar to what researchers have already seen from telaprevir. Patients in the trial reported cases of … Next Page »

Though FDA approval may not happen until the end of this year or at all, is this drug available outside of the United States which is always slow in approving new drugs. Someone I love has HEP C and had not responded to the current treatment. Please help with a direction